Ionis To Get Head Start On Arrowhead With Tryngolza Launch

The antisense oligonucleotide specialist is ready to launch its rare disease therapy Tryngolza but could face competition from a more potent rival by mid-2025.

Ionis HQ
Roche will lead the Phase III development of the therapy, based on Ionis' antisense drug platform. • Source: Ionis

Ionis has gained US Food and Drug Administration approval for Tryngolza, making it the first treatment in the US for the rare metabolic disease familial chylomicronemia syndrome (FCS), and giving it a head start over rival Arrowhead.

The US go-ahead was granted on 19 December, and Tryngolza (olezarsen) will be used an adjunct to diet to reduce triglycerides in adults with FCS, a genetic condition where individuals...

Key Takeaways
  • Ionis has made its name by discovering major rare disease drugs them licensing them out, such as Spinraza to Biogen.
  • Tryngolza is...

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